Fsma.org

Balancing Life’s Tough Times ™
Contacts:Paratek Pharmaceuticals, Inc.Families of SMA
Libertyville, IL and Boston, MA, May 17, 2006 —Families of Spinal MuscularAtrophy (FSMA) and Paratek Pharmaceuticals, Inc. today announced a jointcollaboration to develop a promising drug candidate for the treatment of SpinalMuscular Atrophy (SMA), the leading genetically inherited cause of death of childrenunder the age of two years. The collaboration will focus on optimizing and advancinginto the clinic a novel small molecule within Paratek’s library derived from thetetracycline class of compounds.
Spinal Muscular Atrophy is an often-fatal genetic disorder resulting from the loss ofboth copies of the Survival Motor Neuron (SMN1) gene. This causes a chronicdeficiency in the production of the SMN protein, which is essential to the properfunctioning of the motor neurons in the spinal cord and to the control of muscles inthe limbs, neck and chest.
“This new drug development collaboration will focus on optimizing the drug featuresof a newly identified lead compound that directly influences SMN2 gene splicing,”said Dr. Jill Jarecki, FSMA Research Director. “FSMA-sponsored research hascontributed to the identification of the SMN1 gene as well as a second disease-modifying copy of the gene called SMN2. Normally, the SMN2 gene producesreduced amounts of SMN protein due to a defect in mRNA splicing. This projectaims to develop a SMA drug that safely and effectively restores the proper amount ofSMN protein in the body in order to slow or reverse the disease process by correctingthe splicing of SMN2 gene.”
“Paratek is excited about this collaboration for several reasons. First, we already havea compound with validated activity in hand,” said Dr. Ken Tanaka, Vice President ofResearch and Development for Paratek. “In addition, this project is of great scientific
interest, as the compound has been shown to directly regulate SMN splicing andshould lead to a greater biological understanding of the underlying disease process.With our chemistry expertise and the splicing assay capabilities at Cold SpringHarbor, we believe we have the right resources and team in place to lead us to a noveldrug for treating SMA.”
The Krainer laboratory at Cold Spring Harbor will be assessing additional Paratekcompounds from Paratek’s collection for effects on SMN2 splicing. In parallel,Paratek will be developing new proprietary compounds based on this initial lead. Inrelated work, the FSMA scientific advisory board has recently awarded Dr. MichelleHastings of Cold Spring Harbor Laboratory a two-year grant to investigate themolecular mechanism of this class of compounds.
“FSMA is excited to be funding this drug discovery effort,” said Audrey Lewis,Executive Director of FSMA. “We believe that this compound will work differentlyfrom the other SMN-increasing compounds that FSMA is developing, providing ourcommunity with an additional pathway to potentially treat this disease.”
About Paratek Pharmaceuticals, Inc.
Paratek Pharmaceuticals, Inc. is engaged in the discovery and commercialization ofnew therapeutics that treat serious and life-threatening diseases, with a particularfocus on the growing worldwide problem of antibiotic resistance. Paratek isadvancing novel compounds that can circumvent or block bacterial resistance.Paratek’s lead compound, MK-2764/PTK 0796, is a broad spectrum antibiotic withoral and IV formulations for the treatment of the most common community andhospital bacterial infections, including those caused by resistant strains such asMRSA. This project is being advanced in collaboration with Merck & Co., Inc. Inaddition to its tetracycline-derived antibacterials, Paratek is developing smallmolecule drugs that can prevent infection by interfering with Multiple AdaptationalResponse (MAR) mechanisms in bacteria.
Outside the antibacterial therapeutic area, Paratek has also established an effort toexploit its novel tetracycline derivatives and their unique mechanism of action inselected anti-inflammatory and neurodegenerative conditions, including acollaboration to develop novel non-antibacterial tetracycline derivatives for multiplesclerosis with Serono SA. Paratek has an active chemical synthesis effort to producenovel and diverse small molecules, with the goal of developing non-antibacterialcompounds with improved activity in serious inflammatory and neurodegenerativediseases based upon a growing body of clinical and basic research supporting thisapproach.
Paratek is privately held and headquartered in Boston, Massachusetts, USA. For moreinformation, visit Paratek's website at
About Families of SMA
FSMA is the largest international organization dedicated solely to eradicating SMAby promoting and supporting research in both the private and public sector, helpingfamilies cope through informational programs and support, and educating the public
and the medical community about SMA. The organization, originally founded in 1984by small group of parents, has grown to more than 32 chapters and affiliatesworldwide and more than 5,000 member families and is a founding member of theInternational Alliance for Spinal Muscular Atrophy. FSMA receives the majority ofits funding through volunteer efforts, funding over $25 million to date, and continuesto increase its funding commitments each year with $15 million in new researchplanned over the next three years. For more information visit the website00-886-1762.